Pharnext
SA (Paris:ALPHA) (FR0011191287 - ALPHA), a
biopharmaceutical company pioneering a new approach for the development
of innovative drugs based on the combination and repositioning of known
drugs, today announced successful intermediate analyses for its Phase
III clinical trial, PLEO-CMT, in Charcot-Marie-Tooth Disease Type 1A
(CMT1A).

PLEO-CMT is a pivotal, multi-center, randomized, 15 months, double
blind, placebo-controlled Phase III study that was initiated in December
2015 and has enrolled 323 patients with mild-to-moderate CMT1A in 30
sites across Europe, the U.S. and Canada. As the study will be completed
at the end of 2018, two intermediate analyses, a blind variability
analysis followed by a futility analysis, were therefore carried out as
planned.

According to Professor Philippe Lehert, member of the Independent Data
Safety Monitoring Board (DSMB), the variability of tests between
patients is indeed within predefined limits. In addition, the futility
analysis concludes that PLEO-CMT is sufficiently powered to detect an
effect of PXT3003 on the primary efficacy endpoint. These two analyses
follow two favorable intermediate safety analyses delivered by the DSMB
in November 2016 and September 2017 and indicate therefore that PLEO-CMT
can continue according to the original plan without having to increase
the trial size.

Pr. Daniel Cohen, M.D., Ph.D., Co-Founder and Chief Executive Officer
of Pharnextsaid « These two analyses are additional steps
successfully reached for the PLEO-CMT study. They allow us to confirm
the schedule for final results by the second half of 2018. »

About the Data Safety Monitoring Board (DSMB)
The DSMB is an
independent body of experts drawn from the fields of clinical medicine,
biostatistics and study methodology, chartered to provide
recommendations to Pharnext upon regular pre-specified review of the
accumulated data during the conduct of the clinical trial.

About Pharnext
Pharnext is an advanced clinical-stage
biopharmaceutical company founded by renowned scientists and
entrepreneurs including Professor Daniel Cohen, a pioneer in modern
genomics. Pharnext has two lead products in clinical development.
PXT3003 is currently in an international Phase III trial for the
treatment of Charcot-Marie-Tooth disease type 1A and benefits from
orphan drug status in Europe and the United States. PXT864 has generated
positive Phase 2 results in Alzheimer’s disease. Pharnext is the pioneer
of a new drug discovery paradigm: PLEOTHERAPY™. The Company identifies
and develops synergic combinations of repositioned drugs at new optimal
lower doses. These PLEODRUG™ offer several key advantages: efficacy,
safety and intellectual property including several product or
composition of matter patents already granted. The Company is supported
by a world-class scientific team.